Miller Colton M, Harris Edward N
Department of Biochemistry, University of Nebraska - Lincoln, 1901 Vine St. Lincoln NE 68588 USA.
RNA Dis. 2016;3(4). doi: 10.14800/rd.1393. Epub 2016 Aug 15.
The clinical applicaton of antisense oligonucleotides (ASOs) is becoming more of a reality as several drugs have been approved for the treatment of human disorders and many others are in various phases in development and clinical trials. ASOs are short DNA/RNA oligos which are heavily modified to increase their stability in biological fluids and retain the properties of creating RNA-RNA and DNA-RNA duplexes that knock-down or correct genetic expression. This review outlines several strategies that ASOs utilize for the treatment of various congenital diseases and syndromes that develop with aging. In addition, we discuss some of the mechanisms for specific non-targeted ASO internalization within cells.
随着几种药物已被批准用于治疗人类疾病，并且许多其他药物正处于不同的研发和临床试验阶段，反义寡核苷酸（ASO）的临床应用正日益成为现实。ASO是短的DNA/RNA寡核苷酸，经过大量修饰以提高其在生物体液中的稳定性，并保留形成可敲低或纠正基因表达的RNA-RNA和DNA-RNA双链体的特性。本综述概述了ASO用于治疗各种先天性疾病和随年龄增长而出现的综合征的几种策略。此外，我们还讨论了细胞内特定非靶向ASO内化的一些机制。
